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Saturday, October 24, 2026Conference Co-ChairsUma Borate, MBBSAlice Mims, MDAplastic Anemia/PNH Agenda 7:30 - 8:15 Breakfast and Registration8:15 - 8:45 Welcome 8:45 - 9:15
Bone Marrow Failure
Bone Marrow Failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are…
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Relapse is the leading cause of allogeneic hematopoietic stem cell transplant (allo-HSCT) failure in myeloid neoplasms [1]. Patients receiving reduced-intensity conditioning (RIC) regimens have a higher risk of post-transplant relapse compared to those undergoing myeloablative conditioning (MAC) [2]. However, many patients are not candidates for MAC due to age, comorbidities, or frailty.…
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ABSTRACTIntroductionCentral venous catheter (CVC) insertion is frequently required in patients with haematological diseases. Despite limited evidence,
thrombocytopenia
thrombocytopenia:
(throm-buh-sie-tuh-PEE-nee-uh) A condition in which there is a shortage of platelets in the bloodstream. This results in a low platelet count. Bleeding and bruising…
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AbstractMyeloproliferative neoplasms (MPNs) were previously thought to result solely from somatic mutations; however, familial clustering and germline genetic testing have revealed hereditary MPNs. These hereditary MPNs are one type of hereditary hematopoietic malignancy (HHM) syndrome, or hereditary blood cancer, and generally display an autosomal-dominant inheritance pattern…
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IntroductionPayers employ health care utilization–management strategies, including prior authorization (PA), to limit costs and promote evidence-based, guideline-concordant care. However, PA may be inconsistently applied and associated with patient confusion and frustration.1-3 Patient engagement in PA processes can add to administrative burden and may be associated with adverse…
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Background: Philadelphia-negative myeloproliferative neoplasms (MPNs), including essential thrombocythaemia (ET), polycythaemia vera (PV), and myelofibrosis (MF), usually affect older adults, but a subset occurs in adolescents and young adults (AYA). Regional age-stratified data remain limited.
Methods: We retrospectively studied Philadelphia-negative MPN patients managed at the National Center…
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Highlights -Considerable heterogeneity exists in germline testing implementation across hematopoietic stem cell transplantation centers in the United States.-Lack of standardized protocols affects the timing of testing and inconsistent sample collection workflows.-Barriers to consistent care include insurance challenges, logistical constraints, and…
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Weill Cornell Medicine investigators have developed a new AI-based method to assess patients with a form of blood cancer called myelodysplastic neoplasms. The method, published June 30 in Leukemia, compares the physical locations and morphometric (size and shape) characteristics of every hematopoietic cell in the patient’s
bone marrow
bone marrow:
The soft, spongy tissue inside most…
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To the editorU2 small nuclear RNA auxiliary factor 1 (U2AF1) is one of the most frequently mutated genes in myelodysplastic neoplasms (MDS) [1, 2]. U2AF1 mutations predominantly occur at two hotspots (S34, Q157) within two zinc finger domains and disrupt pre-mRNA splicing by altering RNA binding specificity, with S34 and Q157 variants affecting nucleotide recognition at the −3 and +1 positions of…
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Patients with
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
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paroxysmal nocturnal hemoglobinuria…
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CONFERENCE LOCATION:Heldrich Hotel 10 Livingston AvenueNew Brunswick, NJ 08901Parking: Complimentary hotel valet parking CONFERENCE AGENDA
Aplastic Anemia
Aplastic Anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek…
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The timing of TP53
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone marrow.
acquisition carries important prognostic implications for patients with myeloproliferative neoplasms (MPNs) and…
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Abstract
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Background: Advances in hematopoietic stem cell transplantation (HSCT) have been driven by progress in
supportive care
supportive care:
Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low…
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Haploidentical
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
transplantation (haplo-BMT) with post-transplant
cyclophosphamide
cyclophosphamide:
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your…
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Summary
This UK-based retrospective analysis describes real-world treatment patterns and outcomes in 175 patients with accelerated (AP, n = 69) or blast-phase (BP, n = 106) ‘Philadelphia-negative’ myeloproliferative neoplasms (MPN-AP/BP) diagnosed between 2013 and 2025. Median age at transformation was 71 years. With a median follow-up of 45.2 months, median overall survival (OS) was 14.9 months…
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Correspondence
The Myelodysplastic/Myeloproliferative neoplasm (MDS/MPN) overlap syndromes are a heterogeneous group of rare hematologic malignancies characterized by features of both MDS such as morphologic dysplasia and cytopenias and MPNs including elevated blood counts and often splenomegaly [1]. These syndromes frequently harbor recurrent mutations, underscoring their interrelated biological…
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ABSTRACT
Allogeneic hematopoietic cell transplantation (allo-HCT) is an effective treatment for patients with high-risk hematologic malignancies. Over the last decade, gut microbiota composition during allo-HCT has been associated with patients' outcomes. Treatment-related factors, in particular the use of broad-spectrum
antibiotics
antibiotics:
The most common white blood cells are…
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Recorded Sessions:
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
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DEADLINE MAY 8Senators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a bipartisan letter to the Senate in support of increased funding for the Department of Defense’s medical research accounts, including CDMRP and BMFRP.You can help by asking Your Senators to Sign the LetterSenators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a…
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